Orna Collaborates with Vertex to Develop Gene Therapies for Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT)

Shots:

• Orna & Vertex have entered into a 3yrs. strategic research collaboration to utilize the former’s LNP delivery solutions for supporting the development of next-gen. gene editing therapies for SCD & TDT, with Vertex funding the partnership
• As per the terms, Orna will get $65M upfront (incl. investment in the form of a convertible note), up to $635M as preclinical, research, development, regulatory & commercial milestones plus net-sales-based tiered royalties
• Moreover, Orna is also entitled for an option fees & milestones of ~$365M per candidate for up to 10 additional products, if Vertex exercises its option of collaboration expansion to other indications

Ref: Orna Therapeutics | Image: Orna Therapeutics & Vertex Pharmaceuticals

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